Shots:

• The P-IIIb ASCEND study will evaluate the clinical outcomes & safety of the higher doses of nusinersen in 135 patients aged 5-39yrs. with SMA prior treated with Evrysdi at MRD (5mg). Patients will receive 2 loading doses of nusinersen (50mg- 2wks. apart)- followed by a maintenance dose of 28mg- q4mos. in study period & 1st patients are expected to be enrolled in 2021
• The study will also evaluate upper limb fine motor function in patients aged ≥13yrs. using Konectom app & neurofilament levels as a biomarker. The therapy demonstrated a sustained efficacy & safety profile
• Nusinersen has marketed as Spinraza with the US FDA approved dose (12mg) & is being evaluated in an ongoing DEVOTE study

o­ | Ref: Globe Newswire | Image: Biogen